Neurogene has struck deals to fund its pipeline of gene therapies into the second half of 2026, agreeing to merge with the floundering, Nasdaq-listed Neoleukin Therapeutics and persuading investors to pump $95 million into the combined company.
Former Wall Street analyst Rachel McMinn, Ph.D., founded Neurogene and led it to a $68.5 million series A round and a $115 million series B financing. Since disclosing the series B round late in 2020, the gene therapy specialist has advanced treatments for Rett syndrome and CLN5 Batten disease toward readouts. Preliminary data from phase 1/2 clinicals trials are due in the second half of next year.
The latest agreements will ensure Neurogene can reach and pass those milestones. Through the deals, the biotech will secure Neoleukin’s Nasdaq listing and remaining money plus $95 million from investors including Great Point Partners, EcoR1 Capital and Redmile Group.
Neurogene expects to exit the transactions with around $200 million to its name, making it one of the better funded companies in a gene therapy space that suffered as public investors soured on high-risk, loss-making biotechs.
While Novartis dropped its Rett gene therapy AVXS-201 in 2021, Taysha Gene Therapies is continuing to advance a rival candidate to Neurogene’s NGN-401 through the clinic. Taysha and Neurogene are both using AAV9 vectors to deliver payloads designed to overcome the mutations in the MECP2 gene that drive Rett syndrome.
Neurogene is making the early running in CLN5 Batten disease. While companies including Amicus Therapeutics and Regenxbio have launched studies of genetic therapies against other forms of Batten, Neurogene is the first company to file a CLN5 study with ClinicalTrials.gov.
The decision to merge with Neoleukin to support the gene therapies continues the biotech circle of life. Neoleukin gained its Nasdaq listing through a reverse merger with Aquinox Pharmaceuticals in 2019, but its prospects nose-dived as internal and external clinical data dampened enthusiasm for IL-2. The biotech laid off 70% of its staff and began looking for strategic alternatives in March.